Mitochondria are the energy centers for most eukaryotic cells. They function by converting macronutrients into energy (ATP) which cells can then use. Mitochondria have been implicated in several human diseases and disorders, including mitochondrial disorders, autoimmune disease, cancer, metabolic disease, cardiac dysfunction and heart failure, among others. Key to mitochondrial function is having enough NAD+ in cells, which is used to generate energy for cells and to regulate cell functions such as signaling, immune regulation and cell death.
Mitochondrial myopathy is the term for diseases and disorders arising from damaged or defective mitochondria in cells. For most patients suffering from mitochondrial diseases, no treatments are currently available to treat the underlying deficient mitochondrial function; they are limited to treating symptoms and not the disease itself. There is still a significant unmet need for therapies which can treat mitochondria-related diseases and disorders.